A Single-Center Experience With 1400 Patients

To assess whether the pattern of presentation and the outcome of monoclonal gammopathy of undetermined significance (MGUS) have changed over the last 3 decades, we evaluated 1400 patients, divided into 3 groups: group I (1975-1987), group II (1988-1997), and group III (1998-2007). We observed a significant increase in age (p = 0.001), IgM and biclonal MGUS (p = 0.003), hemoglobin (p < 0.0001), and albumin (p = 0.0001), and a significant reduction of monoclonal (M)-protein concentration (p < 0.0001), percentage of bone marrow plasma cells (p < 0.0001), and beta2-microglobulin (p = 0.0001) over the 3 decades. The proportion of patients with M-protein <1.5 g/dL was significantly higher in group III (66%) than in group II (44%) and group I (26%) (p < 0.0001). By Kaplan-Meier analysis, group III had a significantly lower 5-year probability of transformation (5%) compared to group II (12%) and group I (22%) (p = 0.003). Patients with M-protein <1.5 g/dL had the same life expectancy as the general population (standardized mortality ratio 1.09; p = 0.41). In conclusion, we found that the pattern of presentation of MGUS has changed over time and now includes a higher proportion of patients with more favorable presenting features and probably a better outcome compared to patients presenting in the past. This changing scenario calls for revising the current concepts of the clinical significance of MGUS and the management of patients

The Economic Burden of Multiple Myeloma. Definition of a Model for Forecasting Patients’ Costs

Background: The aim of this study was to evaluate healthcare costs in a single-centre population of patients with multiple myeloma (MM), in an attempt to develop a model for forecasting costs. Methods: A cohort of 387 MM patients, diagnosed at Policlinico San Matteo (Pavia, Italy), between 2002 and 2014, was analysed grouping patients into those eligible (n=223) or not eligible (n=164) for transplantation. After descriptive statistics, the benchmark model - Ordinary Least Squares - and different variations of the Generalized Linear Model were adopted. Results: The average total cost per patient was around €28,500 for patients not eligible for transplantation and around €87,000 for the eligible ones. The difference in marginal costs for transplant-eligible patients was probably due to higher costs for hospitalisation and the costs of the transplant procedure itself. The analysis highlighted four determinants useful for building a model to forecast expenditure: age, bortezomib use, lenalidomide use, and number of lines of therapies. The two most important determinants of expenditure were use of the novel agents and the total number of lines of therapy, which reflects a higher number of doses and a greater need for accesses to hospital. Conclusion: In conclusion, using a Generalized Linear Model, we identified four determinants in our cohort which were useful for building a model to predict expenditure for MM patients. Although the analysis was performed in a particular setting in a single hospital, the model could be applied to any scenario of patients

Elotuzumab plus pomalidomide and dexamethasone in relapsed/refractory multiple myeloma: a multicenter, retrospective real-world experience with 200 cases outside of controlled clinical trials

In the ELOQUENT-3 trial, the combination of elotuzumab, pomalidomide and dexamethasone (EloPd) proved a superior clinical benefit over Pd with a manageable toxicity profile, leading to its approval in relapsed/refractory multiple myeloma (RRMM), who had received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI). We report here a real-world experience of 200 RRMMs treated with EloPd in 35 Italian centers outside of clinical trials. In our dataset, the median number of prior lines of therapy was 2, with 51% of cases undergoing autologous stem cell transplant (ASCT) and 73% exposed to daratumumab. After a median follow-up of 9 months, 126 patients stopped EloPd, most of them (88.9%) because of disease progression. The overall response rate (ORR) was 55.4%, in line with the pivotal trial results. Regarding adverse events, our cohort experienced a toxicity profile similar to the ELOQUENT-3 trial, with no significant differences between younger (&lt;70 years) and older patients. The median progression-free survival (PFS) was 7 months, shorter than that observed in the ELOQUENT-3, probably due to the different clinical characteristics of the two cohorts. Interestingly, the ISS stage III (HR:2.55) was associated with worse PFS. Finally, our series's median overall survival (OS) was shorter than that observed in the ELOQUENT-3 trial (17.5 versus 29.8 months). In conclusion, our real-world study confirms EloPd as a safe and possible therapeutic choice for RRMM who received at least two prior therapies, including lenalidomide and a PI

The place of thalidomide in the treatment of multiple myeloma

Thalidomide represents the first attempt in multiple myeloma (MM) patients to overcome resistance to chemotherapy through a biological agent. The exciting results reported in the first study by Singhal et al. in 1999 [1] led to several other studies which aimed to evaluate its efficacy in different settings and disease phases, to define its toxicity, and to establish the optimal dose. Some of these questions have already been answered while others, such as the best dosage or the best schedule to obtain the highest efficacy with the lowest toxicity, still remain. Thalidomide has been studied as a single agent or in association with other drugs (dexamethasone, chemotherapy and new drugs) showing a synergic activity. We review the results of the main studies on the efficacy and toxicity of thalidomide used as a single agent or in association with other drugs, reflect on its present role, and consider its future contribution to the treatment of MM

Correction: Long Term Evaluation of the Impact of Autologous Peripheral Blood Stem Cell Transplantation in Multiple Myeloma: A Cost-Effectiveness Analysis.

BackgroundHigh-dose therapy with autologous peripheral stem cell transplantation represents today the standard approach for younger multiple myeloma patients. This study aimed to evaluate the long term economic impact of autologous transplantation with respect to conventional therapy.MethodsWe retrospectively reviewed the charts of multiple myeloma patients diagnosed at our department between 1986 and 2003 and treated according to the therapy considered standard at the time of diagnosis. Analysis of costs was done by assessing resource utilization and direct costs were measured and monetized before proceeding with the analysis, based on public health service tariffs.ResultsGroup A including 78 patients treated with Melphalan and Prednisone was compared with Group B including 74 patients who received an autologous transplant. The median overall survival was 3.2 and 5.4 years respectively (p = 0.0002). Mean cost per patient was significantly higher in group B with respect to group A (102373€ vs 23825€; pConclusionsThe cost of autologous transplantation remains high. The calculated incremental cost-effectiveness ratio, however, given the significant prolongation of overall survival obtained with autologous transplantation, is within an acceptable threshold. Notwithstanding, its high cost should be taken into account when considering the whole cost of multiple myeloma